The 41st Symposium: “Making PPI More Accessible: Patient and Citizen Participation in Healthcare You Can Start Tomorrow”

Seminar Date: Tuesday, March 5, 2024

Video Streaming Period: September 30, 2024 (Mon) – September 30, 2025 (Tue) [1 year]

Opening Remarks

In recent years, various stakeholders and organizations have been working to promote PPI (Patient and Public Involvement), and it is now widely recognized that patient involvement is necessary not only in research but also in all areas, including healthcare policy.Pharmaceutical companies are also promoting initiatives to incorporate patients’ voices at every stage—from drug research through to post-marketing—and are working alongside patients to realize better healthcare. I hope this will be an opportunity for us to reflect on PPI together and serve as a catalyst for you to take that first step.

Presentation

演題①　PPI の最新動向~PPIの更なる促進に向けて～
The Office of Pharmaceutical Industry Research 主任研究員 吉田 晃子 氏

PPI is a concept that was first adopted in the United Kingdom. It is often translated into Japanese as “patient and citizen participation.” While the definition was originally specific to the research field—meaning research conducted together with or by patients and citizens—in recent years, the concept has evolved beyond specific research fields to encompass healthcare policy and other areas in general, calling for the participation of patients and citizens in decision-making processes. There is a growing recognition that patients are not merely recipients of established medical care, but partners in shaping better healthcare.
For pharmaceutical companies, using PPI to achieve the best possible outcomes for patients and their families is becoming a fundamental principle.As for why PPI is necessary for pharmaceutical companies, I believe there is a common understanding within the industry that, precisely because a gap exists between the perspectives and needs of pharmaceutical companies and those of patients and their families, it is impossible to create medicines that are truly valuable to patients unless their genuine needs are identified at an early stage.

Survey results show that over 90 percent of patients believe PPI activities are meaningful. However, awareness stands at about 60 percent, and participation is limited to about 10 percent; we hope to see further increases in both awareness and participation in the future.

PPI is a rapidly growing field in global pharmaceutical innovation that is attracting increasing attention. In many other countries, governments have established systems to listen to patients’ voices and promote patient participation. I believe such initiatives are also needed in Japan.While awareness of PPI among patients in Japan is still limited, opportunities to participate in PPI initiatives—not only through the pharmaceutical industry but also through events organized by patient groups and academic societies—are increasing significantly, making it easier than ever to get involved. I strongly encourage you to participate actively.
In the pharmaceutical industry, we aim to understand the true needs of patients and their families, develop medicines that provide value to them at an earlier stage, and work toward realizing a better healthcare environment.

Presentation 2: Expectations for “Participation”—The Perspectives of Those Directly Affected
Ms. Matsuyo Kamata, Representative Director, Public Interest Incorporated Association “Association for People with Dementia and Their Families”

I joined the Family Association in 1990 because I wanted to learn more about dementia. I have served as the representative director since 2023. I was originally a nurse working at a university hospital, but when my father-in-law suffered a cerebral hemorrhage and was left with paralysis on his left side and higher brain dysfunction, I resigned from my job to care for him.At that time, I truly realized how difficult it is to provide care at home, so after my children were grown, I worked in the field of home nursing, as well as at day care centers and special nursing homes.
In 2004, my father at my parents’ home in Saga was diagnosed with Alzheimer’s disease, and four years later, my mother was also diagnosed with the same condition.Following my father’s diagnosis, I commuted long distances every month to provide care for 11 years. During that time, I was also caring for my mother-in-law in Kyoto at home after she was diagnosed with Alzheimer’s disease. Thus, I am both a family caregiver and a healthcare professional.

Our family support group is a public interest incorporated association that was founded in January 1980 and is now in its 44th year. We have 9,400 members, primarily consisting of patients and their families, but anyone interested in dementia is welcome to join.

The very foundation of our family support group’s activities is our regular gatherings. In addition to caregivers sharing their personal caregiving experiences, we host these events as forums for exchanging information—including practical tips from other family caregivers and insights from doctors and other professionals—to help make caregiving a little easier and create a more comfortable environment for the person with dementia. We have continued to hold these gatherings for 44 years.Reports in our newsletter from that time describe how meeting others facing similar caregiving challenges—and realizing there were people struggling even more than themselves—brought relief and gave them courage. Even today, in an era overflowing with information about dementia and caregiving services, the pain of having a family member with dementia can truly only be understood by those who share the same experience. We also publish a newsletter, offer telephone counseling, and conduct dementia awareness campaigns.

Also, as part of our activities, we submit petitions to the Minister of Health, Labor, and Welfare. I believe this is the very foundation of PPI.We have now submitted more than 70 such petitions to the government. After submitting each petition, we hold discussions with government representatives and officials from the Ministry of Health, Labor and Welfare to explain our actual circumstances. However, it has long been a one-way process—essentially just receiving acknowledgments that our requests have been noted.

On January 1 of this year, the Basic Act on Dementia, aimed at promoting the realization of an inclusive society, came into effect. Article 3, Paragraph 3 of the Act states: “Ensuring opportunities to express opinions on matters directly related to oneself and to participate in activities in all sectors of society.” I believe we can equate this “participation” with the expectations placed on PPI.I’m hopeful that, through the efforts of family support groups to convey the voices of those living with dementia, we’ve moved beyond the one-way “we’ve heard you” approach that companies and government agencies have taken in the past, and have now created a space for two-way dialogue.

However, I also have anxieties regarding “participation.” First of all, the term “PPI”—just seeing that Romanized spelling alone makes me very resistant. It makes me feel like I’m being told something complicated.I’m also anxious because I don’t understand what “participation” actually entails or what I’m supposed to do. I don’t know the difference between “participation” and “involvement,” and I’m anxious about being asked to participate in something I don’t fully understand. I lack expertise in pathological mechanisms or the mechanisms underlying the disease, so even if I receive a preliminary explanation, I worry whether I’ll ever be able to truly understand it.

We have very high hopes for this participation. We believe it will guide those currently affected—and those who may be affected in the future—toward better lives. We understand that those affected also need to learn, but they cannot participate without understanding technical terms and medical concepts, so we look forward to support from experts and businesses.By connecting what they learn to their own experiences, participation will improve, and I look forward to a “togetherness” where solutions are created and continuously evolve in collaboration with those directly affected. Through participation, I hope that the experiences of those directly affected will lead to social contribution and a sense of purpose, and that dialogue will foster mutual understanding and synergy among all stakeholders, ultimately improving the lives of patients and their families.

Presentation 3: The Hope of a Small Patient Group—Thoughts on PPI
Ms. Ayako Hiroishi, MLC Patient Group

I work as a company employee, and my husband and I primarily run the patient support group together. We have a daughter in first grade and a 5-year-old son. Three years ago, when our son was 2 years old, he was diagnosed with subcortical cystic megalencephalic leukoencephalopathy (MLC), which is why we started the patient support group.Due to the condition, his head is large and he sometimes stumbles; it’s also said that head trauma can accelerate the progression of the disease, so he wears a headguard. However, he lives a lively and happy life and attends daycare. He’s a perfectly normal 5-year-old boy who laughs a lot.

Major characteristics of MLC include macrocephaly (an enlarged head), developmental delays, intellectual disabilities, and epilepsy. It’s said to be a condition that leads to wheelchair use in the teens, but symptoms and the rate of progression vary from person to person.My son had developmental delays and was attending a developmental support center, but just as he finally started walking at age 2, his primary care physician told me, “This is a disease that will leave him in a wheelchair by his teens.” I simply couldn’t accept it at the time; I was unable to face reality. Even so, those were hellish days where I had to keep moving forward.

MLC is one of the nationally designated intractable diseases—specifically, a form of progressive leukodystrophy—and is also designated as a “chronic specific disease in children.” We have asked Dr. Toshiyuki Yamamoto of Tokyo Women’s Medical University to serve as our patient association advisor. It is estimated that there are currently about 20 people in Japan who have been diagnosed with MLC.

A paper published four years ago by the University of Barcelona on MLC gene therapy served as the starting point for our patient group’s activities. That paper gave us hope—the hope that this might be a treatable disease, but one that is simply unknown and under-researched at present—and inspired us to make sure as many researchers as possible become aware of this condition.

Based on our experiences so far, I’d like to highlight a few challenges we’ve encountered from the patient’s perspective.
In our case, there was no patient support group when we were diagnosed.We reached out to representatives of other established patient groups for rare diseases to hear their stories, and through their introductions, we gradually expanded our network. As part of this process, Dr. Nishikawa from the NPO AASJ—whom we were introduced to by a member of another patient group—agreed to host an MLC study session, and Dr. Yamamoto from Tokyo Women’s Medical University, who serves as an advisor to our patient group, also participated.I often think it would have been helpful to have an organization like this to help us gather information right after our diagnosis. However, since we were still in a tense state—struggling to accept reality—I also believe that gathering information would have been meaningless unless someone was just as desperate as we were to find answers. We continue to gather information to this day.
Regarding the challenges we faced when launching the patient group, we would appreciate advice from pharmaceutical companies on what criteria must be met for the group to be officially recognized, as well as what information we should collect with an eye toward future clinical trials and PPI activities.
Additionally, I believe it would be beneficial to have a system that facilitates matching between disease research and researchers.

Next, regarding the challenges I currently face, the first concerns patient registries. While the specific nature of registries likely varies by disease, I feel that this is precisely where collaboration with researchers, doctors, and pharmaceutical companies is essential.I’ve also heard that the data held by patient organizations offers a high degree of flexibility and is easy to use. Furthermore, as a patient organization, I feel we need to learn what steps are necessary to proceed to clinical trials.
The second challenge is raising awareness. It’s true that focusing solely on raising awareness for MLC isn’t enough—the number of people affected is too small for it to be effective.Furthermore, I’d like to raise awareness not only about MLC but also about leukodystrophy and other rare diseases similar to MLC. I want to do something to help the mothers who must provide medical care every few hours. I’m also wondering how we can secure funding for research in Japan, given that there are large foundations in Europe and the United States.

Furthermore, as science, technology, and medical technology continue to advance, clarifying what was previously unknown, I believe the number of patients with rare diseases will continue to increase. To ensure that these individuals do not lose hope in this country, I hope that, even from the perspective of a small patient organization, we can make a meaningful contribution to PPI.In his presentation, Mr. Yoshida mentioned that PPI helps expand our network of connections, and I agree. I truly believe that PPI isn’t all bad.

Presentation 4: How Should We Approach PPI?
~Let’s Convey the Voices of Patient Organizations~
Ms. Miho Goto, Representative Director, NPO CNS Network Council

I run patient and family support groups focused on mental health and neurological conditions through the CNS (Central Nervous System) Network Council.
The reason I run a family and patient support group specializing in psychiatry is that, when I was a teenager, I lost a close relative of the same age to a mental illness. I was deeply shocked that, despite being so close to them, I had failed to recognize their mental distress, and this left me with a lingering sense of unease—which ultimately led me to major in nursing in college.
　While studying nursing, I became actively involved in family support group activities, and my university graduation thesis was also on the topic of family support groups. Afterward, I worked as a nurse at a psychiatric hospital, and since 2007, I have been working as a clinical trial coordinator and clinical research coordinator at a medical institution involved in drug development.In 2014, I formalized the activities I had been volunteering for—including support groups for people with mental illness, patient groups, and family support groups—into an organization called the CNS Network Council. Currently, I serve as a director of the Institute for Medical Development Infrastructure, a council member of the Clinical Trial Support Foundation, and the chairperson of the Japan SMO Association (which supports the conduct of clinical trials at Investigator Sites).
　Regarding the questions “What is PPI?” and “What does patient and citizen participation actually involve?”, our organization does not specialize exclusively in PPI. However, I would like to share our experiences—including moments when we’ve wondered, “Is this PPI?”—as they arise in our day-to-day activities.
When it comes to patient and citizen participation, I don’t think we need to launch a major initiative right away. Instead, I’d like to convey that we should start by sharing what we’re already doing—not just within our own organization, but also reaching out to the outside world. One way to do this, for example, might be for our organization to reach out to government agencies or pharmaceutical companies.For example, it’s quite common for consumer perspectives and opinions to be incorporated into general products and services, but what about drug development? I think it would be good to share our opinions a bit more.
When engaging in PPI, I think it’s fine to follow the process of learning first and then making our voices heard.
　As for learning, I think that—as tips for organizations looking to advocate with government agencies and pharmaceutical companies, as well as for managing patient groups and ensuring the sustainability of their activities—rather than focusing solely on PPI, we need to learn how to develop and sustain an organization in a way that ensures its long-term viability.As part of developing this literacy, I also think it’s important to learn about health and medical care as it relates to the conditions we or our family members have. In a world where it’s easy to access a wide variety of information online, I feel it’s essential to develop the ability to discern which information is accurate and beneficial for us.

Once you’ve learned more, I believe it’s important to speak up and share your thoughts and opinions.This means sharing concerns and anxieties with other patients and family members, discussing medications, conveying expectations to government agencies, and sharing ideas with pharmaceutical companies—such as suggesting what products would be helpful or expressing thoughts on clinical trials. I also think we need to consider whether holding events online is truly beneficial for patients and their families.

We often talk about how we should approach PPI—that it would be great to have an open dialogue where everyone—patients, families, and myself—feels comfortable, with a “I’m okay, and you’re okay too” kind of relationship.I emphasize that it’s incredibly important to express ourselves honestly, listen carefully to others, and communicate in a way that values both ourselves and the other person. I also stress that fostering open dialogue—including within patient and family support groups—is crucial.
When you hear the term “PPI,” you might feel a bit overwhelmed, wondering where to start or how to approach it. But I think the key is not to overcomplicate things—just start with what you can do and take that first step. That’s what really matters.

Panel Discussion

Ikezaki (hereinafter: titles omitted)

I myself live with a rare neurological disease and currently serve as the secretary-general at PPeCC (P-Peck); I have been active in the field of rare diseases for many years.PPeCC supports people with intractable and chronic diseases, as well as patient advocacy groups, and engages in outreach to society.
You spoke to us earlier about making PPI more accessible to everyone, but from here on, I’d like to hear a bit more about the specifics.Ms. Yoshida, you’ve shared a great deal about the current situation in Japan and examples from overseas. PPI is about patient and citizen participation, and with pioneering examples in the field of cancer, the groundwork for PPI is finally taking shape, and public interest seems to be growing. In light of this, what do you think is necessary to further engage citizens in PPI?

Yoshida

As someone who works for a pharmaceutical company, I’m also just a member of the public when I go home. First and foremost, I think it’s important for everyone to take an interest and participate in various opportunities; I believe that’s where one’s role comes into play. If people share their experiences with family or friends, and those people then start participating as well, I think that circle will continue to expand.Today, many of you used words that convey a sense of mutual understanding, collaboration, and connection—terms with broad implications—and I believe that is what PPI is all about. Especially when it comes to involving the general public, I really hope many people will participate, and I look forward to seeing that network of people grow and expand.

Ikezaki

Terms like “dialogue” and “collaboration” might sound a bit grand, but I realized that simply sharing information about diseases and medications with people close to us is also a form of PPI.Ms. Kamata, your organization is already making policy recommendations, and you mentioned having both expectations and concerns regarding participation in drug development. If you have any specific suggestions—such as “if this were in place, it would make the process more accessible” or “this is what’s needed”—I’d appreciate hearing your thoughts.

Kamada

I used to wonder, “Is it really appropriate for patients to be involved in decisions about medication?” While doctors have approached me about clinical trials, I feel that information on other topics simply isn’t reaching me. I realize we need to take the initiative to seek out information ourselves, but for people living with dementia—who are already struggling just to get by—and their families, accessing that information is simply impossible. I think it would be wonderful if there were a platform that could bridge that gap and make that information more accessible.I think it’s necessary to start by bridging that gap—by delivering that information to places where patients and their families can easily access it.

Ikezaki

I think it’s truly difficult to seek out information on your own while living with an illness. I believe that having access to information close at hand will bring PPI closer to reality. Ms. Hiroishi, based on your own experience, I imagine you had to feel your way through many things, so thank you for sharing how you’ve been blazing a trail.You also mentioned reaching out to various organizations led by people with similar experiences, and you suggested that connecting with researchers will become increasingly important going forward. Could you elaborate on that in more detail?

Hiroishi

If there were doctors who were truly committed to helping patients with these conditions and their families, I believe that alone would be a huge source of relief for those families. I think patients would surely feel encouraged just by the presence of such people, and while I realize this is truly difficult, my hope is that policies, organizations, and systems like this will eventually be established.

Ikezaki

I believe matching is crucial for the hope that life from tomorrow onward might change. Mr. Goto possesses a wealth of diverse insights and experiences within his organization, and I think his message—that we should begin sharing these with the outside world, even if we start small—resonated deeply with everyone here today.Regarding open dialogue, I recall that in Mr. Kamata’s remarks, he mentioned that some people feel hesitant to speak with professionals because they lack the necessary knowledge. I believe this is a key requirement for equal dialogue and a challenge we’ll face as we promote PPI moving forward. If you have any thoughts on this, I’d love to hear them.

Goto

When running patient or family support groups, I emphasize that in order to speak on an equal footing with healthcare professionals and other stakeholders, members should first learn about their own condition, thoroughly understand information such as the medications they are taking, and strive to accurately convey their symptoms to the doctor within the limited time available during a consultation. We actually encourage members to compile this information into a “personal notebook.”I believe that by participating in patient and family support groups and listening to the opinions of various people, we can gain information, deepen our understanding, and learn.

Ikezaki

Regarding the topic of “Patient and Citizen Participation in Healthcare—Steps You Can Take Starting Tomorrow,” which is also today’s subtitle, I realize that it might be difficult for everyone listening today to start taking action immediately tomorrow. However, I believe many of you are hoping to take home some practical tips on what you can actually do.I’d love to hear from all of you—from the perspective of what’s actually feasible and practical—about things you’d like to try or suggestions on what you could put into practice.

Yoshida

From the perspective of the pharmaceutical industry, I believe opportunities like this are extremely important. I also think it’s important for all of us—myself included—to discuss our experiences with PPI and share them with the outside world. I also believe it’s important to hear about everyone’s experiences.

Ikezaki

As Mr. Hiroishi mentioned, I believe that when everyone shares information based on their own experiences and from their own perspectives, this leads to the practical implementation of PPI. What are your thoughts, Mr. Kamata?

Kamada

First of all, since we’re an organization made up of people directly affected by this issue, if we’re told that such support services are being set up for patients and their families—or that this is the direction things are heading—we can pass that information along immediately.Another point is that doctors and pharmacists are often the first point of contact, so if the people in those settings let us—the patients and our families—know that these kinds of opportunities exist, and if that information comes to us from the sources closest to us, then we’ll think, “Ah,"Oh, that’s right—times have changed," and that medicines aren’t just created by experts, but by patients working together with them. I thought it would be great if we could somehow gain that understanding. To achieve that, I believe we need to convey the message—to doctors, pharmaceutical companies, and our own members—that these opportunities exist and that the times have indeed changed.

Ikezaki

Ms. Hiroishi, what do you think?

Hiroishi

I do have a strong desire for the people around me—including large organizations and pharmaceutical companies—to hear my thoughts, but I also believe it’s important to let the people close to me know about this and to talk to those I can confide in. When I shared my thoughts on social media, I found that there were indeed many people who reached out to me. I’ve come to realize how incredibly important these connections are.

Ikezaki

These are indeed actions we can put into practice not just starting tomorrow, but even today. I’d also like to ask Mr. Goto—since you’ve already shared many practical examples in your slides—if you have any ideas or specific actions that everyone listening here can start doing tomorrow, I’d very much like to hear them.

Goto

I’ve been talking about learning and communicating, but I’ve come to realize just how important it is to share our message outwardly. If we keep what our organization is doing confined to our own group—without sharing it externally through social media or other channels—people won’t know what we’re actually doing. At the same time, I think it would be great if pharmaceutical companies also shared information about best practices so we can keep each other updated.

Ikezaki

I hope that not only patient organizations but also pharmaceutical companies and various other stakeholders can share their practical insights.We’ve heard from all of you on the theme of making PPI more accessible and identifying actions you can take starting tomorrow. While everyone’s approach to PPI may differ, I would be very happy if today’s presentations and panel discussion helped you feel even a little closer to PPI and inspired you to try taking some action.

Information Session

Introduction to the “Association for Creating a Society Where Everyone Has Easy Access to Clinical Trials”

Regarding the “Association for Creating a Society Where Everyone Has Easy Access to Clinical Trials” (hereinafter referred to as “the Association”), which was launched in June 2023, we introduced the co-founders and observers, as well as the activities carried out from its inception to the present.Specifically, regarding jRCT*, a database of clinical trial information, challenges have been identified by both those searching for information and those entering it. The background for the Association’s establishment was to urge the Ministry of Health, Labor and Welfare to modify the system to make it user-friendly for all stakeholders.In December 2023, the Association submitted a request for system improvements to the Ministry of Health, Labor and Welfare, and some of the requests submitted by the Association have already been implemented as part of the system updates in January 2024.
The “Create Association” plans to continue holding meetings and submitting requests for system improvements, and we will also continue to explore ways to raise awareness and disseminate information regarding clinical trials.
*Japan Registry of Clinical Trials

Key Takeaways and Closing Remarks

Yoshiyuki Ishida, Executive Director of the Japan Pharmaceutical Manufacturers Association, offered words of appreciation, stating, “While there are many challenges to improving the healthcare environment, it is also important for patient organizations, healthcare professionals, and pharmaceutical companies to collaborate and work together to solve each issue one by one. To that end, I hope we can deepen our mutual understanding through forums like this.”

Informal Networking

After the main program concluded, we set aside time for the speakers, representatives from patient organizations in the audience, and members of the JPMA Drug Evaluation Committee to engage in a free-flowing online discussion. There was a lively exchange of opinions on topics that hadn’t been fully covered and issues people wanted to hear more about.

(Yasuhiro Marumoto, Leader of the Mutual Understanding Task Force, Patient Cooperation Committee)

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